Superior efficacy of CsA plus ATG over CsA monotherapy in pediatric transfusion-independent moderate aplastic anemia

Data regarding the management of transfusion-independent moderate aplastic anemia (MAA) in pediatric patients remains scarce. This study was conducted to address this research gap.

We performed a prospective randomized controlled trial enrolling 29 children with transfusion-independent MAA. Patients were randomly assigned to receive either cyclosporine (CsA) monotherapy (n = 14) or CsA combined with anti-thymocyte globulin (ATG) (n = 15).

Baseline characteristics were well balanced between the two groups. The CsA+ATG group exhibited a higher overall response rate (ORR: 80% vs. 50%) and a significantly higher complete response rate (CRR: 60% vs. 7.1%, P = 0.005) compared with the CsA monotherapy group. At 12 months post-treatment, the combination group demonstrated better hematologic recovery. The 36-month event-free survival (EFS) rate was higher in the combination group than in the monotherapy group (86.7% ± 8.8% vs. 32.5% ± 18.5%), though this difference did not reach statistical significance (P > 0.05), likely due to the small sample size. Shorter pre-treatment disease duration correlated with better response (P = 0.045). Adverse events in both groups were mild to moderate and clinically manageable.

CsA combined with ATG is superior to CsA monotherapy for pediatric patients with transfusion-independent MAA, with a favorable safety profile. Early initiation of immunosuppressive therapy (IST) within 3 months of diagnosis may correlate with better treatment response.

In this study, 29 pediatric patients with transfusion-independent MAA were randomly assigned to two treatment arms (CsA monotherapy, n = 14; CsA+ATG, n = 15). Key findings include: (1) a significantly higher CRR in the combination group (60% vs. 7.1%, P = 0.005); (2) a more than two-fold higher 36-month EFS rate in the combination group (86.7% vs. 32.5%); (3) mild-to-moderate, manageable adverse events in both groups. (4) Shorter pre-treatment disease duration was associated with favorable treatment response (P = 0.045). This study fills the gap in the treatment of transfusion-independent MAA and provides a basis for clinical decision-making.